Ontology highlight
ABSTRACT:
SUBMITTER: Duncan GA
PROVIDER: S-EPMC6054694 | biostudies-literature | 2018 Jun
REPOSITORIES: biostudies-literature
Duncan Gregg A GA Kim Namho N Colon-Cortes Yanerys Y Rodriguez Jason J Mazur Marina M Birket Susan E SE Rowe Steven M SM West Natalie E NE Livraghi-Butrico Alessandra A Boucher Richard C RC Hanes Justin J Aslanidi George G Suk Jung Soo JS
Molecular therapy. Methods & clinical development 20180322
Diffusion of the viral vectors evaluated in inhaled gene therapy clinical trials to date are largely hindered within airway mucus, which limits their access to, and transduction of, the underlying airway epithelium prior to clearance from the lung. Here, we discovered that adeno-associated virus (AAV) serotype 6 was able to rapidly diffuse through mucus collected from cystic fibrosis (CF) patients, unlike previously tested AAV serotypes. A point mutation of the AAV6 capsid suggests a potential m ...[more]